The objective of this research plan is to transition the development of the UltraMag ventricular assist system, which is designed to support neonates and small children, from the preclinical to the clinical trial phase. The work will culminate in a single center, prospective, nonrandomized, open-label, clinical trial. The 2001 report of the NHLBI) sponsored Task Force on Research in Pediatric Cardiovascular Disease identified the pressing need for novel systems to support children with severely compromised cardiac disorders. The pediatric ventricular assist device under development by Levitronix and its development partners, the Texas Heart Institute (THI) and the University of Pittsburgh (UoP), is intended to fulfill the need for acute to chronic mechanical circulatory life support for up to 6-months in infants and small children. Our goal will be to create a viable clinical product for infants and small children, based on the core magnetically levitated technology developed by Levitronix. Uniquely, this small centrifugal pump has no seals, bearings, or other movable parts that are traditionally associated with thrombus formation or mechanical failure. In Phase I of the project, we demonstrated the soundness of this technology for the pediatric application through a series of in vitro and in vivo experiments. In Phase II of the project, we optimized the pump design to accommodate the hemodynamic conditions typical for the pediatric patient, and demonstrated effective hemodynamic performance and biocompatibility in a series of in vivo implants simulating typical clinical conditions. Acknowledging the importance and clinical need of advancing this technology rapidly into the clinical arena, we propose here a Phase II Continuation Grant to support the completion of the hardware development to its final clinical form, performance of a series of final pre-clinical animal validation studies using clinical- grade hardware, followed by initiation of the clinical trial under an FDA approved investigational device exemption (IDE).
Our specific Aims i nclude:
Aim 1 : Demonstrate device endurance and biocompatibility in a juvenile ovine model to confirm readiness for clinical trials providing support up to 180 days. Physiological performance and biocompatibility of the blood pump, control systems, and cannulae will be assessed.
Aim 2 : Conduct """"""""preliminary clinical safety and probable benefit"""""""" trial for up to 180 days of the proposed system in 8 children (ranging from neonate to children weighing up to 40 kg) with decompensated congestive cardiomyopathy. The study will consist of a single-site trial to assess survival, hemodynamic effect, possible thrombogenesis, device reliability, reversal of end-organ dysfunction, and potential for weaning andrecovery. The ultimate goal is to gather sufficient data to convince FDA to allow continuation of the trial to prove safety and probable benefit en route to an HDE. Levitronix intends to financially support the clinical trial costs beyond the first eight patients until FDA approval is obtained.
The UltraMag system is being developed to address the need to support children with cardiomyopathies who face the constant threat of death due to heart failure. The pediatric ventricular assist device is intended to fulfill the need for temporary cardiac life support (up to 180-days) in infants and small children.